Rare Diseases Ireland
RDI
Rare Diseases Ireland (RDI) is the national alliance for rare disease patient organisations in Ireland, working across all rare diseases to improve the lives of the estimated 300,000 people living with rare diseases in Ireland.
ID: 309832251878-17
Lobbying Activity
Response to EU Life sciences strategy
9 Apr 2025
The EU life sciences strategy must give hope to the millions of people living with rare diseases in Europe and around the world. We have reached a tipping point as regards new innovative medicines, devices and technologies, including big data and AI. This strategy must ensure that member states embed these innovations into their healthcare systems. We know that research and innovation drives better healthcare outcomes - we must embed research and innovation into our healthcare systems across all member states to drive better outcomes for all. By so doing we will become an attractive location for future research, trials and investment which will help to drive the establishment of more start up companies and licensing/commercialisation by established industry. For too long we have relied on other countries to develop and invest in new technologies and innovative products - Europe must now lead from the front in this regard. Europe could and should do much more to ensure that all countries have equitable best-in-class healthcare systems. Major challenges for People Living With Rare Diseases (PLWRD) include: 1. Being heard and included in all aspects of development, prioritisation and implementation of this planned life sciences strategy. PLWRD (and their representative organisations) must have a seat at the table - we are partners in this work, not outsiders. No one has more invested in the success of this strategy than those who stand to benefit with improved health and quality of life. 2. Access to new medicines - all medicines should be available in all countries at, or close to, the same time. We must strive to eliminate the challenges and disparities in access between different countries. Access is bot the same as reimbursement - there needs to be some type of risk-sharing model established to ensure those that need a medicine are not denied because of the country that they live in. This is particularly so for PLWRD when there may only be a handful of people living with a particular rare disease in smaller member states. 3. Uniform access to screening across member states, especially newborn/early childhood screening. All European citizens should receive their diagnosis as quickly as possible. It is not acceptable for some to have permanent impairment because of a failure to diagnose early in their country of birth. 4. Access to expertise, knowledge and research is critical for PLWRD. The EU has established European Reference Networks (ERNs) to enable this. ERNs must become the engine to drive research into rare diseases. Collaboration between industry and ERNs also needs to be formalised to drive the best possible outcomes for PLWRD. 5. Data and registries are the foundation upon which new technologies and innovations in rare diseases are developed. The EHDS is thus a critical component of this strategy that must drive research and innovation while protecting the privacy of individuals. We welcome a new EU Lifesciences Strategy and seek to support its development and implementation for the benefit of all EU citizens and in particular those living with rare diseases.
Read full responseResponse to Evaluation and revision of the general pharmaceutical legislation
8 Nov 2023
Rare Diseases Ireland is the national alliance for rare disease patient organisations in Ireland. As such we are concerned about innovation and the development and approval of medicines across all rare diseases, and timely access for people living with rare disease to those medicines. We are happy to read that specific objectives of this revision include the provisions to: make sure all patients across the EU have timely and equitable access to safe, effective, and affordable medicines; and offer an attractive innovation-and competitiveness friendly environment for research, development, and production of medicines in Europe. Orphan medicines have transformed the lives of people living with a small number of rare diseases. We want to see similar benefits afforded to people living with other rare diseases, people for whom there may be little hope today. The failure to bring significantly more OMPs to market over the last twenty years must however be considered a failure of the ecosystem in place today. This revision to the legislation and regulations must be designed to drive significant further investment into development of orphan medicines that meet unmet needs, as defined by those with lived experience of rare diseases. Incentives that bring about more innovation and faster development and approval of new and transformative OMPs addressing UMN and HUMN are only useful if they are coupled with access to these medicines across the EU. There is a worrying gap in the legislation to address this well documented challenge. Delays in being able to access new OMPs are multi-factorial. An EU wide system should be adopted with equitable access for all as its core mission. Access to medicines is a topic that is particularly fraught and emotive, bringing people living with rare diseases onto the streets in protest in Ireland. We would like to see the EU doing as much as possible to alleviate the pressure points in the system and eliminate the need for this vulnerable population having to protest on the streets in order to get access to a medicine that is freely available to people living with the same rare diseases in other EU member states. The burden on individuals living with rare diseases, their family members and society are enormous. Every effort must be made to address the unmet needs of people living with rare diseases, as defined by people with lived experience, and future-proof ourselves against the evolving geo-politics of medicines investment and innovation. The proposed revisions to the legislation are welcome however they fail to drive sufficient innovation or solve problems inherent across the ecosystem, with respect to putting patients and their lived experience at the centre of innovation and the challenges associated with access/reimbursement systems today. Significant further changes will be required if we are to address the slow rate of rare disease medicines development, reverse the trends with the EUs deteriorating position as regards medicines innovation and address the market access challenges that exist today. More must be done to favour therapeutic areas with no existing treatment options, be this through development of a new active substances or re-purposing well-established substances. Focused work and innovation in the area of rare diseases will enhance the lives of the many that have been left behind and have not felt the benefit of innovation in medicines and healthcare that has been made over the last 20-30 years for more common conditions. Joined-up thinking under the umbrella of a European Action Plan will help to drive innovation towards those underserved areas of high unmet medical need. We are looking to the European Union to take this opportunity of revision of this legislation to create a robust framework that will truly drive innovation and improve access to transformative treatments for our underserved population of rare disease patients in Ireland over the years and decades to come.
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